<p><strong>BACKGROUND: </strong>Using patient-reported outcomes for symptom monitoring in oncology has resulted in significant benefits for adult patients with cancer. The feasibility of this approach has not been established in the routine care of children with cancer.</p>

<p><strong>METHODS: </strong>The Pediatric Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (Ped-PRO-CTCAE) is an item library that enables children and caregivers to self-report symptoms. Ten symptom items from the Ped-PRO-CTCAE were uploaded to an online platform. Patients at least 7&nbsp;years old and their caregivers were prompted by text/email message to electronically self-report daily during a planned hospitalization for chemotherapy administration. Symptom reports were emailed to the clinical team caring for the patient, but no instructions were given regarding the use of this information. Rates of patient participation and clinician responses to reports were systematically tracked.</p>

<p><strong>RESULTS: </strong>The median age of the participating patients (n&nbsp;=&nbsp;52) was 11&nbsp;years (range, 7-18&nbsp;years). All patients and caregivers completed an initial login, with 92% of dyads completing at least 1 additional symptom assessment during hospitalization (median, 3 assessments; range, 0-40). Eighty-one percent of participating dyads submitted symptom reports on at least half of hospital days, and 54% submitted reports on all hospital days. Clinical actions were taken in response to symptom reports 21% of the time. Most patients felt that the system was easy (73%) and important (79%). Most clinicians found symptom reports easy to understand and useful (97%).</p>

<p><strong>CONCLUSIONS: </strong>Symptom monitoring using patient-reported outcome measures for hospitalized pediatric oncology patients is feasible and generates data valued by clinicians and patients.</p>

<p>Patient reported outcomes (PROs) are the gold standard for assessing patients' experience of treatment in oncology, defined in the 21st Century Cures Act as information about patients' experiences with a disease or condition, including the impact of a disease or condition, or a related therapy or clinical investigation on patients' lives; and patient preferences with respect to treatment of their disease or condition [1]. PROs provide a comprehensive assessment of the benefits and risks of new medical products, as well as essential data to inform real-world use. Although RCTs are the ultimate source for information for evaluating products in development, they are not always feasible for rare diseases with few or no effective treatment options available. Thus, it is important to consider other measures that can help to improve the strength of evidence for cell and gene therapies targeting rare indications. While collection of PROs and other patient experience endpoints does not resolve the difficulty of conducting trials in small populations, doing so contributes empirical evidence that informs both product development and patient access. Additionally, including routine collection of PROs in registries may provide supplemental data to further characterize the benefit:risk profile of cell and gene therapies at follow-up times that would be infeasible to operationalize in a clinical trial setting.</p>

<p>Symptom monitoring using patient-reported outcomes (PROs) is not common in pediatric oncology, despite interest from stakeholders-including patients, families, clinicians, and regulatory organizations-and proven clinical benefit in adult oncology. This article examines the foundational data for patient-reported symptom reporting in this population and posits the next investigative steps toward the implementation of patient-reported symptom monitoring in the care and research of pediatric oncology patients. The reasoning behind, and feasibility of, monitoring symptoms in pediatric oncology patients using PRO measures are discussed, as well as specific tools that have been developed to track symptoms in this population, including innovative electronic self-reporting platforms built to engage children in the symptom reporting process. Aspects of engaging both patients and clinicians in the symptom self-report process are reviewed, as are the experiences of "early adopters" of this process in pediatric oncology and across pediatrics. It is clear that there are key issues that remain regarding the use of PROs for symptom monitoring, including selection of specific outcomes to monitor, how to resolve discrepant reports, and determination of benefit. The next steps for investigation of these issues are discussed. Unanswered questions notwithstanding, work should continue to make patient-reported symptom monitoring an established, evidence-based part of routine and research practice in pediatric oncology.</p>